Expanded Access or Compassionate Use

FDA regulations use the term "Expanded Access" although you may also see the terms "Compassionate Use" or "Treatment Use". Expanded Access regulations provide a mechanism to use or administer an unapproved medical device or investigational drug outside a clinical trial.

Submissions for all categories of Expanded Access require sponsors to include adequate information to demonstrate that such access to the drug or device will not interfere with its clinical investigations or product development. The FDA must determine that access to the investigational product for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the Expanded Access use, or otherwise compromise its potential development.

Investigational Devices

Expanded Access for devices includes emergency use and treatment use of an investigational device to treat a patient with a serious or immediately life-threatening disease or condition for whom there are no comparable or satisfactory alternative treatment options.

Investigational Drugs

Expanded Access for drugs permits the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition for whom there are no comparable or satisfactory alternative treatment options.

FDA regulations allow Expanded Access for the use of an unapproved medical device outside of the approved clinical trial parameters. Expanded access for devices includes emergency use and treatment use ("Compassionate Use") of an investigational device to treat a patient(s) with a serious or immediately life-threatening disease or condition for which no satisfactory alternative therapy exists.

FDA regulations allow access to investigational devices for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in the clinical trial. The treatment must be for a serious disease or condition for which no alternative treatment is available to the patient.

For specific information on procedures at Emory see the FDA page at the IRB website.

EMERGENCY USE - the FDA permits the use of an Investigational Medical Device in order to save a patient's life even though there is no current IDE for the device, or the patient does not meet the protocol criteria for the IDE, or the physician or institution is not an approved user/site under the IDE. In this situation, Emory physicians should reference the steps outlined in IRB P & P 66 Emergency Use of Investigational Medical Devices (see link below). Following use, the physician must promptly notify the Emory Institutional Review Board (IRB) and IDE Sponsor, or FDA if there is no IDE sponsor.

TREATMENT USE - the FDA permits an Investigational Medical Device to be used during a clinical study with patients who would appear to benefit from the device but do not meet the requirements for being included in the IDE clinical investigation. In this situation, Emory physicians should reference the steps outlined in IRB P & P 67 Treatment Use of Investigational Devices (see link below). A written Expanded Access IDE supplement must be submitted for review and approval by the FDA prior to use. The treatment use must also be submitted for review and concurrence or approval of the IRB prior to beginning this treatment use of the investigational device for patients.

A licensed practitioner who holds the expanded access IDE and receives an investigational device for treatment use under an expanded access IDE is a "sponsor-investigator" and is responsible for meeting all applicable sponsor responsibilities as well as investigator responsibilities. FDA reporting requirements include submission of progress reports. Expanded Access regulations for devices are contained in Title 21, Code of Federal Regulations, Part 812.36.

FDA uses the term "Expanded Access" for the use of investigational drugs for treatment of patients who are not enrolled as subjects in studies under the IND that covers the drug being studied.  Expanded access, sometimes called "compassionate use", permits the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition for whom there is no comparable or satisfactory alternative treatment options.

FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial.  They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials.

For specific information on procedures at Emory see the FDA page at the IRB website.

EMERGENCY USE: When a patient needs to be treated with an investigational drug before a written IND submission can be completed, the PI must obtain authorization for individual use from FDA by telephone or electronic communication with subsequent submission of IND paperwork and IRB notification.  FDA provides a Physician Fact Sheet (PDF) which covers the steps required and a link to contact information. In this situation, Emory physicians should reference the steps outlined in IRB P&P 70 Investigational New Drugs (PDF).

NON-EMERGENCY USE: In this situation, a written Expanded Access IND must be submitted for review and approval by the FDA prior to use of an investigational drug.  The protocol must also be approved by the Emory Institutional Review Board (IRB) prior to treating a patient.

A licensed practitioner who holds the expanded access IND and receives an investigational new drug for treatment use under an expanded access IND is a "sponsor-investigator" and is responsible for meeting all applicable sponsor responsibilities as well as investigator responsibilities.  FDA reporting requirements include submission of IND safety reports and annual reports.  Expanded Access regulations for drugs are contained in Title 21, Code of Federal Regulations, Part 312.300.

At the conclusion of treatment, the sponsor investigator must provide FDA with a written summary of the results of the expanded access use including adverse effects, and request to withdraw the IND. Click here for the Expanded Access IND Withdrawal template (DOCX).

For additional information see: